MicroRNAs (miRNAs) are single-stranded, non-coding RNAs that regulate transcription and translation of coding RNAs (mRNA). Since their discovery in 1993, miRNAs have emerged as key regulators in numerous physiological and pathological processes. miRNAs are highly conserved and are about 18-25 nucleotides in length. Typically, miRNAs direct translational repression by binding to the 3′ untranslated region (UTR) of mRNAs. Because only partial complementarity is required for miRNA-mRNA interactions, a single miRNA can potentially regulate hundreds of mRNA transcripts.
Motor neuron diseases are those involving progressive loss of structure or function of motor neurons, including death of motor neurons. Such diseases include Amyotrophic Lateral Sclerosis (ALS), primary lateral sclerosis, progressive muscular atrophy, progressive bulbar palsy and pseudobulbar palsy. Most motor neuron diseases have no cure and available therapeutics are targeted at improving symptoms, relieving pain, and slowing degeneration. Motor neuron diseases, such as ALS, need novel, innovative approaches to drug development since many traditional therapeutics have failed or only shown marginal benefits. Further, there are no specific tests to diagnose motor neuron diseases. Early diagnosis or detection of motor neuron disease may facilitate earlier treatment thus slowing degeneration. Thus, in addition to novel drugs, there is a need for early, accurate detection of motor neuron disease.
While miRNAs are known regulators of physiological and pathological processes, little is known about their involvement in motor neuron conditions or diseases. Further, knowledge of motor neuron-specific miRNAs may enhance the detection and treatment of motor neuron diseases. Compositions and methods exploiting motor neuron-specific miRNA regulation in motor neuron conditions or diseases are needed to further medical research and provide diagnostic and therapeutic resources for such conditions and diseases. The present invention provides compositions and methods for detecting and treating conditions and diseases associated with aberrant motor neuron-specific miRNA regulation.